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Effect of Nicotinamide in Friedreich's Ataxia

NCT01589809 · Status: UNKNOWN · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 40

Last updated 2017-07-02

No results posted yet for this study

Summary

The purpose of the interventional study is to determine whether Nicotinamide is effective at upregulating the Frataxin (FXN) gene in patients with Friedreich's ataxia (FRDA) where this gene is abnormally 'switched off'.

The purpose of the non-interventional study is to investigate the use of novel, highly-sensitive technology to capture clinical deficit and measure subtle changes in the activities of daily living and to correlate functional changes to levels of expression of Frataxin protein and the epigenetic structure of the Frataxin gene over a 9-12 month period without nicotinamide. Healthy volunteers will be included as comparators in this part of the study.

Conditions

Interventions

DRUG

nicotinamide

dose-escalation, 2-8 grams, oral

Sponsors & Collaborators

  • Imperial College London

    lead OTHER

Principal Investigators

  • Vincenzo Libri, Dr · Imperial College London

  • Paola Giunti, Dr · NHNN, 02034483153

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
Yes

Timeline & Regulatory

Start
2012-06-30
Primary Completion
2015-06-30
Completion
2017-12-31

Countries

  • United Kingdom

Study Locations

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Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01589809 on ClinicalTrials.gov